Health Care

'Mind-blowing' trial results give hope for haemophilia cure

'Mind-blowing' trial results give hope for haemophilia cure”

Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them.

There are around 2,000 people with severe haemophilia A in the UK.

The condition, which puts sufferers at risk of excessive bleeding even from slight injuries, is now incurable.

The condition can range from mild to moderate to severe depending on the levels of clotting factor VIII in the blood.

The factor serum is made from human blood, or if the haemophilia patient forms antibodies against that they will have to use an artificial recombinant which means genetic materials are brought together to create the desired genetic sequence that enables clotting.

Commenting on the findings in an emailed statement, Liz Carroll, chief executive of The Haemophilia Society, welcomed the results but says gene therapy raises some questions and says it remains unclear how the treatment would affect patients in the long run.

It can also cause life-threatening internal bleeding, which in turn can lead to joint damage and arthritis, and there is no known cure for the condition.

The trial saw patients across England injected with a copy of the missing gene, which allows their cells to produce the missing clotting factor. The trial moved all participants to a stage where they no longer needed regular treatment for the condition, while 11 of them have now got "normal or almost normal" levels of the missing protein factor after being monitored for 19 months.

Professor John Pasi, Haemophilia Centre Director at Barts Health NHS Trust, said: 'We have seen mind-blowing results which have far exceeded our expectations. When we started out we thought it would be a huge achievement to show a five per cent improvement, so to actually be seeing normal or near normal factor levels with dramatic reduction in bleeding is quite simply wonderful. It's usually inherited, and most people who have it are male. "It is so exciting".

Larger trials are set to commence in the coming months to include sufferers in the USA, Europe, Africa and South America. "A single dose of medication that can so dramatically improve the lives of patients across the world is an incredible prospect".

"It's ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing".

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Jake Omer, 29 from Billericay, Essex, who took part in the trial, revealed the gene therapy changed his life and that he now has "hope for the future".

United Kingdom doctors say they have developed a "ground breaking" gene therapy technique that might lead to an effective "cure" for haemophilia A.

The father-of-two was diagnosed with haemophilia when he was two and has had frequent injections of factor VIII to prevent bleeds ever since.

Prior to this study, participants received up to 185 FVIII infusions per year to prevent bleeds, resulting in up to 41 breakthrough bleeding episodes per year despite prophylactic treatment.

In a statement, Jake says: "The gene therapy has changed my life".

In contrast to standard care, which requires multiple intravenous therapy infusions per week, this gene therapy appears to have long-lasting effects after a single infusion.

Jake says he finds it odd no longer having to worry about bleedings or swelling.

The first time he knew it had worked was four months after the therapy when he dropped a gym weight and bashed his elbow.

The trials took place at five United Kingdom sites: The Royal London, Guys and St Thomas', Birmingham, Cambridge and Hampshire hospitals.

He started to panic, but after icing the injury that evening, everything was normal the next day.



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